NICHD Nobel Prize Winners

The NICHD supports quality research and investigation that helps to improve the lives of children, adults, families, and communities. Over the Institute's 50 years, several of our grantees have been awarded the Nobel Prize in recognition for scientific and other advances.

His work allows scientists to engineer mice with mutations in any desired gene. Usually, scientists "knock out" one or more genes with unknown functions through targeted mutations. By observing the animal with the "knocked out" gene, they can infer the missing gene's probable role. Because mice are genetically the closest laboratory animal to humans, this discovery has made "knockout mice" an invaluable animal model, particularly in the field of genetics. The animal models of human diseases that are made with knockout mice are critical for evaluating potential disease treatments before beginning human clinical trials.

Prior to their research there was confusion about how RNA interference occurs and the role it plays. By shutting off specific genes, RNAi exerts a critical influence on the activity of genes and the transmission of genetic information. Fire and Mello found that this RNA interference was unique to the gene whose code matched the injected RNA molecule in C. Elegans. They also discovered that RNAi can spread from cell to cell and can be inherited genetically.

Moreover, Fire and Mello illuminated the critical role of double stranded RNA (dsRNA) in RNAi. They found that tiny pieces of double-stranded RNA (dsRNA) were very effective at shutting down specific genes in C. Elegans. From this observation they deduced that dsRNA plays a seminal role in RNA interference.

Because only small numbers of dsRNA molecules were required for the observed outcomes, Fire and Mello concluded that a catalytic process was involved. Fire and Mello's discoveries were a revelation—they found that RNA interference is a critical mechanism for controlling the flow of genetic information. The use of dsRNA to promote RNA interference has widespread potential clinical applications. For example, genes known to cause high cholesterol were recently "shut off" in animal models by introducing silencing RNA.

Horvitz found specific genes controlling cell death in C. elegans, a roundworm well-suited to genetic analysis. This finding was critical because organisms require billions of cells to die every day, since they also need to manufacture billions of cells per day. This process of genetically directed cell death is called "controlled cell death."

The process of controlled cell death is critical in numerous biological processes such as the remodeling of tissues after degenerative disease or injury. Understanding this process has resulted in improved treatment for healing wounds and tissue damage.

Statistical analyses based on non-random samples can lead to erroneous conclusions and poor policy, but population-based samples are frequently not available. The Heckman correction, a two-step statistical approach, corrects for non-randomly selected samples and allows researchers to determine whether specific findings may apply to broader populations. Much of Heckman's work has been used to help understand how events in early life affect individuals' future earning potential and economic well-being.

Furchgott ascertained that blood vessels become dilated because the endothelial cell produced a hitherto unknown signal molecule, which allows vascular smooth muscle cells to relax. He called this signal molecule the endothelium-derived relaxing factor (EDRF). However, even after Furchgott identified EDRF's role, scientists did not know what type of molecule EDRF was.

Ignarro discovered that EDRF was identical to nitric oxide (NO). This finding had myriad applications for medical treatment. For example, NO gas inhalation has been used to reduce dangerously high blood pressure in the lungs of infants. Ignarro's research also laid the groundwork for the development of erectile dysfunction medications such as Viagra. The researchers found that NO can cause erection by dilating the blood vessels to the erectile bodies.

NO is just one of many "Reactive Oxygen Species" that not only function as signaling molecules in cells but can also induce tissue damage, which prompted the inclusion of anti-oxidants in dietary supplements.

Wieschaus and Nüsslein-Volhard used Drosophila as an animal model to find and catalog genes that are critical for determining the plan and formation of body segments. Lewis studied how genes regulate the development of individual body segments into specialized organs. He established that genes are arranged on the chromosomes in the order of the body segments that they control. For example, the first genes in a complex of developmental genes direct the head region, genes in the middle manage abdominal segments, and the last genes control the tail region.

Wieschaus and Lewis' research has helped to explain the origins of congenital malformations in the human body. Subsequent studies revealed that similar genes were found in humans, which revolutionized the study of human development and developmental disorders.

Becker pioneered studies that applied models of rational behavior to areas once thought to be dictated largely by instinct or irrationality. For example, Becker explored the economic rationale behind the family structure, pointing out that family units produce basic goods such as meals and entertainment.

Becker was one of the first economists to study how individuals and families invest in education and experience to produce human capital, and his research broadened understanding of the factors influencing wages, incomes, and wealth over extended periods of time.

Prior to Altman and Cech's research, enzymes were considered to be exclusively proteins. Enzymes are biocatalysts that increase reaction rates as much as one million-fold. Without biocatalysts, chemical reactions in organisms would seldom take place. Altman and Cech demonstrated that like some proteins, RNA can also act as a biocatalyst. Altman and Cech's research broadened our understanding of RNA; we now know the molecule to be not just a transmitter of genetic information between DNA and proteins, but also a biocatalyst in its own right.

1986: Stanley Cohen & Rita Levi-Montalcini

Cohen isolated the nerve growth factor (NGF) from both snake venom and male mouse salivary glands, which are rich sources of NGF. He injected the isolates from the venom and salivary glands into mice and found that the extracts stimulated non-nerve growth. Cohen attributed this phenomenon to another formerly unknown substance, which he termed the "epidermal growth factor" (EGF).

Cohen and his colleagues went on to illuminate how EGF influences development and provides a target for chemotherapy. This research has proven fundamental to understanding the development of cancer and for designing anti-cancer drugs.

Prostaglandins are made from unsaturated fatty acids, principally arachidonic acid, which resides in the cellular membrane. The membrane itself has the enzymatic capacity to produce prostaglandins. These compounds are released when the tissue function is disturbed by trauma, disease, or anxiety, allowing normal function to continue despite the stresses. Prostaglandins can now be produced synthetically and have many clinical uses including the induction of childbirth, treatment of peptic ulcers, the reduction of pulmonary hypertension, and the treatment of glaucoma.

1977: Roger C.L. Guillemin & Andrew Schally
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Peptide hormones are substances created by short amino acid chains. Guillemin and Schally's work highlighted the importance of these substances for the regulation of growth, development, reproduction, and stress reactions. Several new peptides were isolated from the hypothalamus, including the inhibitor of pituitary function: somatostatin, which decreases production of the pituitary growth hormone.

Their research provided the groundwork for brain hormone research that greatly enhanced the study and treatment of numerous diseases and disorders, including thyroid diseases, fertility problems, and diabetes.

Specifically, de Duve's research led to the discovery of lysosomes, which are critical organelles for breaking down waste and cellular debris. He also identified peroxisomes, which are found in practically all eukaryotic cells, as organelles. The primary function of peroxisomes is to break down long fatty acid chains, which are crucial for maintaining normal function in the brain and lungs of mammals.

Subsequent studies revealed a number of hereditary diseases with lysosomal enzyme deficiencies, where indigestible material in the lysosomes swell and engorge the cell, preventing it from functioning properly. The discovery, understanding and development of treatment for such diseases would not be possible without the seminal research of de Duve, Claude, and Palade.

1972: Gerald M. Edelman & Rodney R. Porter

Prior to Edelman and Porter's research, scientists did not know how ostensibly identical assortments of antibody proteins were able to target a nearly infinite range of foreign objects, such as viruses or bacteria. Edelman and Porter's findings solved this riddle.

They discovered that antibodies are made up of two long, heavy protein chains and two identical shorter, light chains that make three sections, which compose a Y-shaped molecule. At both points atop the "Y" chain, a light strain is paired with a heavy strain of protein to make a discrete binding location for each target. A third section, which links the two chains together, forms the stem of the "Y." This region remains the same from one antibody to another, but the binding sites are unique, allowing them to "specialize" in targeting specific foreign agents in the body.

This discovery provided a foundation for subsequent research on this critical element of the human body's defense system. Edelman and Porter's discoveries were a breakthrough in the study of how antibodies function. Their findings ignited a frenzy of research worldwide that continues to yield practical results for the treatment of numerous diseases and clinical diagnostics.